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Updated: Mar 7, 2026

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Precision medicine needs randomized clinical trials

Everardo D Saad1, Xavier Paoletti2, Tomasz Burzykowski3

  • 1International Drug Development Institute (IDDI), Avenue Provinciale 30, 1340 Ottignies-Louvain-la-Neuve, Belgium.

Nature Reviews. Clinical Oncology
|February 8, 2017
PubMed

Insights

Randomized trials are crucial for validating new cancer drugs, even with promising early results. Uncontrolled trials may overestimate treatment effects, hindering accurate drug and biomarker development.

Area of Science:

  • Oncology
  • Clinical Trials
  • Precision Medicine

Background:

  • Precision medicine is transforming cancer research, leading to numerous new therapies.
  • The approval of effective drugs based on uncontrolled phase I trials is increasing.
  • This trend challenges traditional clinical trial designs and drug approval processes.

Purpose of the Study:

  • To question the reliance on uncontrolled trials for drug efficacy.
  • To highlight the necessity of randomized controlled trials (RCTs) in drug development.
  • To emphasize the role of randomization in validating biomarkers and facilitating seamless development phases.

Main Methods:

  • Discussion of evidence from uncontrolled phase I trials.
  • Analysis of the limitations of single-arm trials in assessing treatment effects.
  • Examination of the role of biomarkers and companion testing in drug development.

Main Results:

  • Uncontrolled trials may overestimate treatment efficacy due to prognostic factors.
  • Randomization is essential to definitively ascertain the predictive role of biomarkers.
  • Expedited drug approval based on uncontrolled trials carries significant disadvantages.

Conclusions:

  • Randomized trials with a true control arm are necessary throughout drug development.
  • Increased use of randomization supports smoother transitions in drug and biomarker development.
  • RCTs are vital for accurate assessment of novel cancer therapeutics and companion diagnostics.

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